Transcript Do now: 5 minute discussion with a partner

What is genetic engineering?
Personal Genetics Education Project (pgEd)
Harvard Medical School
www.pged.org
Do now: 5 minute discussion with a partner:
1. You’ve been offered a deal from a genomics company. You
can get a free genome sequence – an analysis of all your
DNA that includes a report of your ancestry, traits and
medical profile. The medical profile tells you about diseases
for which you have a low risk as well as those for which you
have a high risk. Are you interested? Why?
2. For the first 100 volunteers, the company is offering to
”correct” several of the disease-related genes found by the
analysis. Would you volunteer for this added service? It is
approved by the government for safety, but it is a very new
procedure without a great deal of long term study. (Note:
This service is not available and will not be in the near
future, so use your imagination.)
What is genetic modification?
Repairing liver disease in mice
by deleting and/or adding genes
Image via https://www.flickr.com/photos/121483302@N02
Altering genes that affect the
human nervous system
/
Image via www.flickr.com/carolinabio
Animals – humans
Why might someone want gene therapy?
’Via Flickr/Wellcome Trust -EM Unit, UCL Medical School, Royal Free Campus
https://commons.wikimedia.org/wiki/Category:Cystic_fibrosis#/m
edia/File:Blausen_0286_CysticFibrosis.png
What is CRISPR?
(Clustered regularly interspaced short palindromic repeats)
A genome editing technique that:
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Targets a specific section of DNA
Makes a precise cut/break
Can replace or delete small pieces of DNA
Harnesses cellular mechanisms to insert
engineered DNA
How is this different from what is
traditionally thought of as genetic engineering?
DNA template with
desired sequence
Cas9 nuclease
with guide RNA
DNA base to be changed
DNA edited to
desired sequence
Can genetic engineering reduce malaria?
Infant surrounded by malaria bed net. Ghana. Photo: © Arne Hoel / World Bank Flickr user: World Bank Photo
Collection
Image via https://www.flickr.com/photos/thefinessimo/2164822357
Researchers have used genetic engineering to
cure a type of liver disease in adult mice
Image via https://www.flickr.com/photos/lex-photographic/16744172269
A solution to the global shortage of organs
for those in need?
Photo via: Maidiel1 on wikicommons
2015: Researchers experiment with human embryos
show CRISPR can work to make genetic modifications
Image of 3 day old embryo via wikipedia
What is the path forward?
Safety – ethics – informed consent –
human and ecological health